Genetic medicine can leave people with rare mutations behind. But there’s new hope
By Laura Ungar,
Associated Press
| 04. 26. 2025
Emily Kramer-Golinkoff can’t get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting.
She has the most common fatal genetic disease in the U.S., which afflicts 40,000 Americans. But her case is caused by a rare genetic mutation, so medications that work for 90% of people with cystic fibrosis won’t help her.
The same dynamic plays out in other genetic conditions. Stunning advances in genetic science have revealed the subtle, insidious culprits behind these brutal diseases and have started paving the way for treatments. But patients with these exceedingly rare mutations have fewer options and poorer prospects than those with more typical forms of these diseases — and many are now pinning hopes on experimental gene therapies.
“We feel such pure joy for our friends who have been lifted from this sinking ship,” said Kramer-Golinkoff, 40. “But we just feel so eager and desperate to join them. It’s really hard to be in this minority of people left behind.”
It’s not just science that is working against these patients...
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